Substance / Medication

Migalastat

Overview

Active Ingredient
migalastat
RxNorm CUI
2054252

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Administration details including route, dosing schedules, preparation instructions, and contraindications are pending physician authorship.

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
A Systematic Review on Safety and Efficacy of Migalastat for the treatment of Fabry's Disease.
Majid Haya, Verma Neharika, Bhandari Shivani et al. · Expert Opin Pharmacother · 2024
PMID: 38753367Meta-Analysis
Long-term efficacy of migalastat in females with Fabry disease.
Kallish Staci, Camporeale Antonia, Hopkin Robert J et al. · J Med Genet · 2025
PMID: 40897525TrialFull text (PMC)
Efficacy and safety of migalastat in a Japanese population: a subgroup analysis of the ATTRACT study.
Narita Ichiei, Ohashi Toya, Sakai Norio et al. · Clin Exp Nephrol · 2020
PMID: 31889231TrialFull text (PMC)
Safety of switching to Migalastat from enzyme replacement therapy in Fabry disease: Experience from the Phase 3 ATTRACT study.
Hughes Derralynn A, Nicholls Kathleen, Sunder-Plassmann Gere et al. · Am J Med Genet A · 2019
PMID: 30920142TrialFull text (PMC)
Migalastat improves diarrhea in patients with Fabry disease: clinical-biomarker correlations from the phase 3 FACETS trial.
Schiffmann Raphael, Bichet Daniel G, Jovanovic Ana et al. · Orphanet J Rare Dis · 2018
PMID: 29703262TrialFull text (PMC)
Treatment of Fabry's Disease with the Pharmacologic Chaperone Migalastat.
Germain Dominique P, Hughes Derralynn A, Nicholls Kathleen et al. · N Engl J Med · 2016
PMID: 27509102Trial

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Risk profile, common and serious adverse effects, contraindications, and drug interactions are pending physician authorship.

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Migalastat (substance)
SNOMED CT
765340000
UMLS CUI
C2698220
RxNorm CUI
2054252

Clinical Data

This intervention maps to 1 entities in the Ltrl knowledge graph.

1
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.